Patient engagement approaches to drive repurposing success: lessons from REMEDi4ALL and Multisulfatase Deficiency

<p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" class="first" dir="auto" id="d16520194e83">Drug repurposing has huge potential to quickly drive therapeutic hypotheses to the clinic and deliver patient impact. This alone makes repurposing an attractive strategy for rare diseases, where there is huge unmet medical need caused by a paucity of treatment options and scientific understanding. Identifying repurposing opportunities in such an understudied environment is challenging, while recruiting and retaining a significant number of patients for clinical trials can be prohibitive. In this environment, patient engagement is central to repurposing success. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d16520194e85">The REMEDi4ALL platform has been developed to have patient input at the heart of its repurposing projects for this very reason. REMEDi4ALL aims to include a patient champion in the heart of our repurposing development teams. These individuals provide vital patient input, guiding project development to ensure a focus on the expressed needs of the patient community, ensuring meaningful impact and real value to society. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d16520194e87">The best example of the impact of this model comes from REMEDi4ALL’s demonstrator project to repurpose tazarotene for the ultrarare multisulfatase deficiency. Working alongside REMEDi4ALL’s patient champion and the United MSD foundation has unlocked a pharmaceutical industry data package to support our project. Furthermore, we have gathered detailed patient perspective data to influence the scientific advice process, answering vital questions from the regulators through patient experience. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d16520194e89">Here, we review the benefits and challenges of REMEDi4ALL’s approach, its impact on our demonstrator project, and our hopes for patient engagement in future repurposing projects.